BPC January 24 update

15 key near-term biotech stock catalysts - likely 1Q 2019

Weekly watchlist

At the beginning of the year we noted a number key data readouts due this year. With the first month of 1Q almost at an end, we highlight key readouts slated by the end of March, including a couple for 1H 2019 that might readout this quarter. It is by no means a full list but these events will be closely watched. Included in the list are four key PDUFA dates from the list noted last week.

First, let’s review the week that was in what was a relatively quiet shortened trading week.

Editas Medicine, Inc. (NASDAQ: EDIT) shares closed Tuesday down 20% to $20.95 following news its CEO, Katrine Bosley, resigned as President and CEO. Shares of other CRISPR companies also fell on the news with CRISPR Therapeutics AG (NASDAQ: CRSP) closing down 11% to $33.08, while shares of Intellia Therapeutics, Inc. (NASDAQ: NTLA) closed down 7% to $14.16.

Bristol-Myers Squibb Company (NYSE:BMY) announced Thursday that following discussions with the FDA it has withdrawn its supplemental Biologics License Application (sBLA) for the Opdivo and low-dose Yervoy for treatment of first-line advanced non-small cell lung cancer (NSCLC). Further data due 1H 2019 from its Phase 3 trial, Checkmate-227, will be required for analysis before resubmitting the application.

AbbVie (NYSE:ABBV) shares closed Friday down 6% to $80.54 following the release of 4Q 2018 financial results. Investors sold down shares of the company on news that its main drug, Humira, saw its international sales decline by 14.8% due to competition from biosimilars.

Advaxis, Inc. (NASDAQ:ADXS) announced Wednesday the FDA has placed its Phase 3 trial of axalimogene filolisbac (AXAL) for the treatment of cervical cancer (AIM2CERV) on partial clinical hold. The hold follows requests for additional information pertaining to certain AXAL chemistry, manufacturing and controls (CMC) matters. All currently enrolled patients will continue to receive treatment, per the trial protocol. However, no new patients can enrol in AIM2CERV until resolution of this partial hold.

A number of biopharma companies announced public offerings of common stock this latest week, while a couple also noted reverse stock splits.

Offerings: BLPH CRBP PTCT RNN SELB URGN WVE NCNA (later withdrawn)

Reverse stock splits: OHRP TTNP

Biotech stock near-term catalysts (likely 1Q 2019):

Drug Stage Catalyst Market Cap

ALNY – Alnylam Pharmaceuticals Inc.
Acute hepatic porphyrias

NDA Filing Rolling NDA completion announced June 5, 2019.
$7.8 billion

GILD – Gilead Sciences Inc.
Selonsertib (GS-4997) - STELLAR 4
Nonalcoholic steatohepatitis (NASH)

Phase 3 Phase 3 trial did not meet primary endpoint - February 11, 2019.
$86.6 billion

ICPT – Intercept Pharmaceuticals Inc.
Ocaliva (Obeticholic acid (OCA)) - REGENERATE
Adult nonalcoholic steatohepatitis (NASH) patients.

Phase 3 NDA filing due 3Q 2019.
$2.3 billion

IMGN – ImmunoGen Inc.
Mirvetuximab soravtansine
Cancer - ovarian cancer and relapsed endometrial cancer

Phase 3 Phase 3 top-line data released March 1, 2019 did not meet primary endpoint. Further Phase 3 trial to be initiated later in 2019 with data due 1H 2022.
$370.8 million

JNJ – Johnson & Johnson
Treatment resistant depression

Approved FDA approval announced March 5, 2019.
$357.7 billion

KPTI – Karyopharm Therapeutics Inc.
Quadruple Refractory Multiple Myeloma

Approved FDA Approval announced July 3, 2019. Continued approval may be contingent upon data from its Phase 3 Boston trial (data due late-2019/early 2020).
$511.3 million

LXRX – Lexicon Pharmaceuticals Inc.
Type 1 Diabetes

CRL CRL issued March 22, 2019.
$631.3 million

MRK – Merck & Company Inc. (new)
KEYTRUDA and Inlyta - KEYNOTE-426
Renal cell carcinoma

PDUFA priority review FDA approval announced April 22, 2019.
$208.4 billion

NVAX – Novavax Inc.
RSV vaccine - prepare trial
Respiratory Syncytial Virus (RSV) in healthy pregnant women - protect infants via maternal immunization

Phase 3 Phase 3 top-line data released March 28, 2019 - primary endpoint not met. Noted June 10, 2019 that a further Phase 3 trial is required.
$110.8 million

PFE – Pfizer Inc.
Duchenne muscular dystrophy (DMD)

Phase 1b Phase 1b data June 28, 2019 noted safety concerns. Phase 3 trial to commence 1H 2020.
$237.7 billion

PRTO – Proteon Therapeutics Inc.
Vonapanitase (PRT-201) PATENCY-2
Chronic kidney disease (CKD) patients undergoing surgical placement of an arteriovenous fistula (AVF)

Phase 3 Phase 3 data released Match 28, 2019 did not meet primary endpoint.
$8.4 million

PTI – Proteostasis Therapeutics Inc.
PTI-428 + PTI-801 + PTI-808
Cystic fibrosis

Phase 2 Phase 2 trial planned with enrolment to be completed by YE 2019.
$50.2 million

REPH – Recro Pharma Inc.
Intravenous (IV) meloxicam
Acute pain following bunionectomy surgery

CRL First CRL announced May 24, 2018. Second CRL issued March 22, 2019.
$224.9 million

SGEN – Seattle Genetics Inc.
Enfortumab vedotin - EV201
Urothelial cancer

BLA Filing Phase 2 pivotal data noted 44% ORR. CR rate 12%. BLA filing due 2019.
$10.3 billion